AR119430A1 - Métodos para tratar o prevenir la atrofia muscular espinal - Google Patents

Métodos para tratar o prevenir la atrofia muscular espinal

Info

Publication number
AR119430A1
AR119430A1 ARP200102019A ARP200102019A AR119430A1 AR 119430 A1 AR119430 A1 AR 119430A1 AR P200102019 A ARP200102019 A AR P200102019A AR P200102019 A ARP200102019 A AR P200102019A AR 119430 A1 AR119430 A1 AR 119430A1
Authority
AR
Argentina
Prior art keywords
antisense compound
muscular atrophy
spinal muscular
treat
methods
Prior art date
Application number
ARP200102019A
Other languages
English (en)
Spanish (es)
Inventor
Kenneth Swope Loveday
Fengju Judy Bai
Lilly East
Wildon R Farwell
Original Assignee
Biogen Ma Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biogen Ma Inc filed Critical Biogen Ma Inc
Publication of AR119430A1 publication Critical patent/AR119430A1/es

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Dermatology (AREA)
  • Food Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
ARP200102019A 2019-07-19 2020-07-17 Métodos para tratar o prevenir la atrofia muscular espinal AR119430A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962876360P 2019-07-19 2019-07-19

Publications (1)

Publication Number Publication Date
AR119430A1 true AR119430A1 (es) 2021-12-15

Family

ID=71995097

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200102019A AR119430A1 (es) 2019-07-19 2020-07-17 Métodos para tratar o prevenir la atrofia muscular espinal

Country Status (31)

Country Link
US (2) US20220325282A1 (fr)
EP (2) EP3999643B1 (fr)
JP (2) JP7279253B2 (fr)
KR (1) KR20220042113A (fr)
CN (1) CN114302727A (fr)
AR (1) AR119430A1 (fr)
AU (1) AU2020316997A1 (fr)
BR (1) BR112022000870A2 (fr)
CA (1) CA3149906A1 (fr)
CL (1) CL2021003602A1 (fr)
CO (1) CO2022001354A2 (fr)
CR (1) CR20220071A (fr)
DK (1) DK3999643T5 (fr)
ES (1) ES2966791T3 (fr)
FI (1) FI3999643T3 (fr)
HR (1) HRP20231515T1 (fr)
HU (1) HUE064071T2 (fr)
IL (1) IL289745A (fr)
JO (1) JOP20220007A1 (fr)
LT (1) LT3999643T (fr)
MA (1) MA62935B1 (fr)
MD (1) MD3999643T2 (fr)
MX (1) MX2022000441A (fr)
PE (1) PE20220941A1 (fr)
PL (1) PL3999643T3 (fr)
PT (1) PT3999643T (fr)
RS (1) RS64879B1 (fr)
SI (1) SI3999643T1 (fr)
TW (1) TWI841762B (fr)
UY (1) UY38790A (fr)
WO (1) WO2021016032A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3797780B1 (fr) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions et procédés de modulation de l'épissage du smn2 chez un sujet
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
FI3999643T3 (fi) 2019-07-19 2023-11-30 Biogen Ma Inc Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi
WO2024138018A1 (fr) * 2022-12-23 2024-06-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Traitement de troubles induits par gemin5

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE027486T2 (en) 2005-06-23 2016-09-28 Isis Pharmaceuticals Inc Preparations and methods for modifying SMN2-Splicing
WO2008157753A1 (fr) * 2007-06-21 2008-12-24 Sma Foundation Procédés de traitement d'une atrophie musculaire spinale
CA2765396C (fr) 2009-06-17 2019-08-20 Isis Pharmaceuticals, Inc. Compositions et methodes pour moduler l'epissage de smn2 chez un sujet
WO2011032109A1 (fr) 2009-09-11 2011-03-17 Sma Foundation Biomarqueurs de l'atrophie musculaire spinale
EP2714037B1 (fr) * 2011-05-25 2016-07-13 Université Paris Descartes Inhibiteurs de la voie erk pour le traitement de l'amyotrophie spinale
EP2943225A4 (fr) * 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc Compositions et procédés pour la modulation de l'épissage de smn2 chez un sujet
BR112017004056A2 (pt) * 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
MX2019000619A (es) * 2016-07-15 2019-08-01 Ionis Pharmaceuticals Inc Compuestos y metodos para la modulacion de smn2.
TW201927241A (zh) 2017-12-21 2019-07-16 瑞士商赫孚孟拉羅股份公司 用於肌肉失能之數位生物標記
MA51678A (fr) 2018-01-25 2020-12-02 Biogen Ma Inc Méthodes de traitement de l'amyotrophie musculaire
IT201800004359A1 (it) 2018-04-10 2019-10-10 IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
FI3999643T3 (fi) 2019-07-19 2023-11-30 Biogen Ma Inc Menetelmiä spinaalisen lihasatrofian hoitamiseksi tai ehkäisemiseksi

Also Published As

Publication number Publication date
CO2022001354A2 (es) 2022-03-18
MD3999643T2 (ro) 2024-02-29
DK3999643T3 (da) 2023-12-04
PT3999643T (pt) 2023-12-11
JP2022540993A (ja) 2022-09-21
BR112022000870A2 (pt) 2022-03-22
WO2021016032A1 (fr) 2021-01-28
TW202116331A (zh) 2021-05-01
AU2020316997A1 (en) 2022-03-10
CN114302727A (zh) 2022-04-08
KR20220042113A (ko) 2022-04-04
SI3999643T1 (sl) 2024-01-31
US20240318182A1 (en) 2024-09-26
DK3999643T5 (da) 2024-08-05
JP2023100911A (ja) 2023-07-19
US20220325282A1 (en) 2022-10-13
HUE064071T2 (hu) 2024-02-28
CR20220071A (es) 2022-04-22
PE20220941A1 (es) 2022-05-31
EP4335503A3 (fr) 2024-06-05
RS64879B1 (sr) 2023-12-29
CA3149906A1 (fr) 2021-01-28
ES2966791T3 (es) 2024-04-24
JOP20220007A1 (ar) 2023-01-30
MA62935B1 (fr) 2023-11-30
EP3999643B1 (fr) 2023-11-22
CL2021003602A1 (es) 2022-10-14
PL3999643T3 (pl) 2024-02-19
HRP20231515T1 (hr) 2024-03-01
JP7279253B2 (ja) 2023-05-22
FI3999643T3 (fi) 2023-11-30
EP4335503A2 (fr) 2024-03-13
TWI841762B (zh) 2024-05-11
MX2022000441A (es) 2022-03-25
IL289745A (en) 2022-03-01
EP3999643A1 (fr) 2022-05-25
LT3999643T (lt) 2023-12-11
UY38790A (es) 2021-01-29

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